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Page 1
Access in the rare diseases landscape.
Jonker AH, Cavaller-Bellaubi M, Nishimura Y, Pearce DA. Jonker AH, et al. Among authors: cavaller bellaubi m. Lancet Glob Health. 2024 Oct;12(10):e1587. doi: 10.1016/S2214-109X(24)00341-3. Lancet Glob Health. 2024. PMID: 39304232 Free article. No abstract available.
SATURN: assessing the feasibility of utilising existing registries for real-world evidence data collection to meet patients, regulatory, health technology assessment and payer requirements.
Sangiorgi L, Boarini M, Mordenti M, Wang V, Westerheim I, Skarberg RT, Cavaller-Bellaubi M, Clancy J, Pinedo-Villanueva R, Lente EJV, Marchetti M. Sangiorgi L, et al. Among authors: cavaller bellaubi m. Orphanet J Rare Dis. 2024 Sep 12;19(1):336. doi: 10.1186/s13023-024-03341-4. Orphanet J Rare Dis. 2024. PMID: 39267100 Free PMC article.
Drug repurposing for rare: progress and opportunities for the rare disease community.
Jonker AH, O'Connor D, Cavaller-Bellaubi M, Fetro C, Gogou M, 't Hoen PAC, de Kort M, Stone H, Valentine N, Pasmooij AMG. Jonker AH, et al. Among authors: cavaller bellaubi m. Front Med (Lausanne). 2024 Jan 17;11:1352803. doi: 10.3389/fmed.2024.1352803. eCollection 2024. Front Med (Lausanne). 2024. PMID: 38298814 Free PMC article.
Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA.
Cavaller-Bellaubi M, Hughes-Wilson W, Kubinová Š, Van de Casteele M, Van Lente EJ, Degortes E, Pontén J, Eichler HG, Le Cam Y, Boselli S, Bucsics A. Cavaller-Bellaubi M, et al. Orphanet J Rare Dis. 2023 Jun 12;18(1):144. doi: 10.1186/s13023-023-02774-7. Orphanet J Rare Dis. 2023. PMID: 37308991 Free PMC article.
Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents.
Croft NM, de Ridder L, Griffiths AM, Hyams JS, Ruemmele FM, Turner D, Cheng K, Lutsar I, Greco M, Gołębiewska Z, Laumond F, Cavaller-Bellaubi M, Elgreey A, Altepeter TA, Pallidis C, Norga K, Nelson R, Crandall W, Vassal G. Croft NM, et al. Among authors: cavaller bellaubi m. J Crohns Colitis. 2023 Mar 18;17(2):249-258. doi: 10.1093/ecco-jcc/jjac135. J Crohns Colitis. 2023. PMID: 36130314 Free PMC article.
Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action.
Cavaller-Bellaubi M, Faulkner SD, Teixeira B, Boudes M, Molero E, Brooke N, McKeaveney L, Southerton J, Vicente MJ, Bertelsen N, García-Burgos J, Pirard V, Reid K, Ferrer E. Cavaller-Bellaubi M, et al. Ther Innov Regul Sci. 2021 Sep;55(5):936-953. doi: 10.1007/s43441-021-00282-z. Epub 2021 May 10. Ther Innov Regul Sci. 2021. PMID: 33970465 Free PMC article.